IL219704A0 - Novel viral vector construct for neuron specific optimized continuous dopa synthesis in vivo - Google Patents
Novel viral vector construct for neuron specific optimized continuous dopa synthesis in vivoInfo
- Publication number
- IL219704A0 IL219704A0 IL219704A IL21970412A IL219704A0 IL 219704 A0 IL219704 A0 IL 219704A0 IL 219704 A IL219704 A IL 219704A IL 21970412 A IL21970412 A IL 21970412A IL 219704 A0 IL219704 A0 IL 219704A0
- Authority
- IL
- Israel
- Prior art keywords
- vivo
- viral vector
- vector construct
- neuron specific
- novel viral
- Prior art date
Links
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
- C12N15/861—Adenoviral vectors
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/0004—Oxidoreductases (1.)
- C12N9/0071—Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14)
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- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
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- A61P25/18—Antipsychotics, i.e. neuroleptics; Drugs for mania or schizophrenia
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- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
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- C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
- C12N9/14—Hydrolases (3)
- C12N9/78—Hydrolases (3) acting on carbon to nitrogen bonds other than peptide bonds (3.5)
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- C12N2750/00011—Details
- C12N2750/00041—Use of virus, viral particle or viral elements as a vector
- C12N2750/00043—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
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- C12N2830/34—Vector systems having a special element relevant for transcription being a transcription initiation element
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- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/40—Vector systems having a special element relevant for transcription being an insulator
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- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
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- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
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- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
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- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/60—Vector systems having a special element relevant for transcription from viruses
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- C12Y—ENZYMES
- C12Y114/00—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
- C12Y114/16—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14) with reduced pteridine as one donor, and incorporation of one atom of oxygen (1.14.16)
- C12Y114/16002—Tyrosine 3-monooxygenase (1.14.16.2)
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- C12Y305/00—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5)
- C12Y305/04—Hydrolases acting on carbon-nitrogen bonds, other than peptide bonds (3.5) in cyclic amidines (3.5.4)
- C12Y305/04016—GTP cyclohydrolase I (3.5.4.16)
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- Health & Medical Sciences (AREA)
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- Bioinformatics & Cheminformatics (AREA)
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- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Health & Medical Sciences (AREA)
- Medicinal Chemistry (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- Pharmacology & Pharmacy (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Psychiatry (AREA)
- Addiction (AREA)
- Virology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Plant Pathology (AREA)
- Immunology (AREA)
- Pain & Pain Management (AREA)
- Psychology (AREA)
- Cardiology (AREA)
- Urology & Nephrology (AREA)
- Heart & Thoracic Surgery (AREA)
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- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (2)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US25950209P | 2009-11-09 | 2009-11-09 | |
PCT/EP2010/067155 WO2011054976A2 (en) | 2009-11-09 | 2010-11-09 | Novel viral vector construct for neuron specific optimized continuous dopa synthesis in vivo |
Publications (1)
Publication Number | Publication Date |
---|---|
IL219704A0 true IL219704A0 (en) | 2012-07-31 |
Family
ID=43466711
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
IL219704A IL219704A0 (en) | 2009-11-09 | 2012-05-09 | Novel viral vector construct for neuron specific optimized continuous dopa synthesis in vivo |
Country Status (15)
Country | Link |
---|---|
US (2) | US20120309816A1 (en) |
EP (1) | EP2498825B1 (en) |
JP (1) | JP5894535B2 (en) |
KR (1) | KR20120089743A (en) |
CN (1) | CN102695526A (en) |
AU (1) | AU2010316982B2 (en) |
BR (1) | BR112012010866A2 (en) |
CA (1) | CA2779437A1 (en) |
CL (1) | CL2012001215A1 (en) |
CO (1) | CO6541559A2 (en) |
IL (1) | IL219704A0 (en) |
MX (1) | MX2012005450A (en) |
NZ (1) | NZ600104A (en) |
RU (1) | RU2606012C2 (en) |
WO (1) | WO2011054976A2 (en) |
Families Citing this family (56)
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US9217155B2 (en) | 2008-05-28 | 2015-12-22 | University Of Massachusetts | Isolation of novel AAV'S and uses thereof |
US8734809B2 (en) | 2009-05-28 | 2014-05-27 | University Of Massachusetts | AAV's and uses thereof |
CA2833905C (en) | 2010-04-23 | 2019-09-10 | University Of Massachusetts | Multicistronic expression constructs |
EP2561073B1 (en) | 2010-04-23 | 2016-08-24 | University of Massachusetts | Cns targeting aav vectors and methods of use thereof |
WO2012135389A2 (en) * | 2011-03-28 | 2012-10-04 | The Regents Of The University Of California | Host cells and methods for oxidizing aromatic amino acids |
ES2661680T3 (en) | 2011-04-21 | 2018-04-03 | University Of Massachusetts | Compositions based on VAAr and methods for treating alpha-1 anti-trypsin deficiencies |
US10072251B2 (en) | 2014-02-19 | 2018-09-11 | University Of Massachusetts | Recombinant AAVS having useful transcytosis properties |
EP4410805A2 (en) | 2014-03-18 | 2024-08-07 | University of Massachusetts | Raav-based compositions and methods for treating amyotrophic lateral sclerosis |
US10689653B2 (en) | 2014-06-03 | 2020-06-23 | University Of Massachusetts | Compositions and methods for modulating dysferlin expression |
US10577627B2 (en) | 2014-06-09 | 2020-03-03 | Voyager Therapeutics, Inc. | Chimeric capsids |
US10370432B2 (en) | 2014-10-03 | 2019-08-06 | University Of Massachusetts | Heterologous targeting peptide grafted AAVS |
US10711270B2 (en) | 2014-10-03 | 2020-07-14 | University Of Massachusetts | High efficiency library-identified AAV vectors |
EP3209311B1 (en) | 2014-10-21 | 2024-03-06 | University of Massachusetts | Recombinant aav variants and uses thereof |
MX2017005834A (en) | 2014-11-05 | 2017-11-17 | Voyager Therapeutics Inc | Aadc polynucleotides for the treatment of parkinson's disease. |
RU2749882C2 (en) | 2014-11-14 | 2021-06-18 | Вояджер Терапьютикс, Инк. | Modulating polynucleotides |
EP3218484A4 (en) | 2014-11-14 | 2018-05-30 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
US11697825B2 (en) | 2014-12-12 | 2023-07-11 | Voyager Therapeutics, Inc. | Compositions and methods for the production of scAAV |
EP3256170B1 (en) | 2015-02-13 | 2020-09-23 | University of Massachusetts | Compositions and methods for transient delivery of nucleases |
WO2016172155A1 (en) * | 2015-04-23 | 2016-10-27 | University Of Massachusetts | Modulation of aav vector transgene expression |
CA3021949C (en) | 2015-04-24 | 2023-10-17 | University Of Massachusetts | Modified aav constructs and uses thereof |
SG10202012832VA (en) * | 2015-05-28 | 2021-01-28 | Univ Cornell | Adeno-associated virus mediated delivery of c1ei as a therapy for angioedema |
US20190032079A1 (en) * | 2015-08-03 | 2019-01-31 | Myodopa Limited | Systemic synthesis and regulation of l-dopa |
CA3002980A1 (en) | 2015-10-22 | 2017-04-27 | University Of Massachusetts | Prostate-targeting adeno-associated virus serotype vectors |
US11253576B2 (en) | 2015-10-22 | 2022-02-22 | University Of Massachusetts | Methods and compositions for treating metabolic imbalance in neurodegenerative disease |
CA3006569A1 (en) | 2015-12-02 | 2017-06-08 | Voyager Therapeutics, Inc. | Assays for the detection of aav neutralizing antibodies |
CA3011939A1 (en) | 2016-02-02 | 2017-08-10 | University Of Massachusetts | Method to enhance the efficiency of systemic aav gene delivery to the central nervous system |
US11060088B2 (en) | 2016-02-12 | 2021-07-13 | University Of Massachusetts | Anti-angiogenic miRNA therapeutics for inhibiting corneal neovascularization |
EP3440210A4 (en) | 2016-04-05 | 2019-11-27 | University of Massachusetts | Compositions and methods for selective inhibition of grainyhead-like protein expression |
US11413356B2 (en) | 2016-04-15 | 2022-08-16 | University Of Massachusetts | Methods and compositions for treating metabolic imbalance |
US11299751B2 (en) | 2016-04-29 | 2022-04-12 | Voyager Therapeutics, Inc. | Compositions for the treatment of disease |
RU2758488C2 (en) | 2016-05-18 | 2021-10-28 | Вояджер Терапьютикс, Инк. | Modulating polynucleotides |
JP7220080B2 (en) | 2016-05-18 | 2023-02-09 | ボイジャー セラピューティクス インコーポレイテッド | Compositions and methods for treating Huntington's disease |
US11882815B2 (en) | 2016-06-15 | 2024-01-30 | University Of Massachusetts | Recombinant adeno-associated viruses for delivering gene editing molecules to embryonic cells |
EP3506817A4 (en) | 2016-08-30 | 2020-07-22 | The Regents of The University of California | Methods for biomedical targeting and delivery and devices and systems for practicing the same |
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AU2017341849B2 (en) | 2016-10-13 | 2024-03-21 | University Of Massachusetts | AAV capsid designs |
WO2018204786A1 (en) | 2017-05-05 | 2018-11-08 | Voyager Therapeutics, Inc. | Compositions and methods of treating amyotrophic lateral sclerosis (als) |
WO2018204803A1 (en) | 2017-05-05 | 2018-11-08 | Voyager Therapeutics, Inc. | Compositions and methods of treating huntington's disease |
US11859179B2 (en) | 2017-05-09 | 2024-01-02 | University Of Massachusetts | Methods of treating amyotrophic lateral sclerosis (ALS) |
GB2562774A (en) * | 2017-05-25 | 2018-11-28 | Mcdonald Michael | Genetic construct |
JOP20190269A1 (en) | 2017-06-15 | 2019-11-20 | Voyager Therapeutics Inc | Aadc polynucleotides for the treatment of parkinson's disease |
KR101956042B1 (en) | 2017-07-13 | 2019-03-08 | 서강대학교산학협력단 | Introducing method of l-dihydroxyphenylalanine in target protein |
JP7229989B2 (en) | 2017-07-17 | 2023-02-28 | ボイジャー セラピューティクス インコーポレイテッド | Trajectory array guide system |
CA3069703A1 (en) * | 2017-07-19 | 2019-01-24 | Steven Hoffman | Compositions for treating stress-related disorders |
EP3662060A2 (en) | 2017-08-03 | 2020-06-10 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
AU2018338188A1 (en) | 2017-09-22 | 2020-04-02 | University Of Massachusetts | SOD1 dual expression vectors and uses thereof |
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EP3697908A1 (en) | 2017-10-16 | 2020-08-26 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
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KR20120089743A (en) | 2012-08-13 |
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CL2012001215A1 (en) | 2012-08-03 |
CN102695526A (en) | 2012-09-26 |
JP5894535B2 (en) | 2016-03-30 |
NZ600104A (en) | 2013-11-29 |
BR112012010866A2 (en) | 2016-11-29 |
WO2011054976A3 (en) | 2011-07-07 |
JP2013509890A (en) | 2013-03-21 |
EP2498825A2 (en) | 2012-09-19 |
CA2779437A1 (en) | 2011-05-12 |
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